Pharma industry backs FDA NGS guidance but seeks clarification on off-target data and manufacturing changes

The pharmaceutical sector has broadly endorsed the FDA's recent guidance on using next‑generation sequencing to evaluate the safety of human gene‑therapy products.

Despite this support, companies are asking the agency to clarify several key points, notably how off‑target sequencing data should be reported and interpreted.

Stakeholders also seek guidance on the appropriate methods for nominating sequences and on what additional information is required when manufacturing processes change.

These clarification requests aim to provide a clearer, more consistent framework for assessing gene‑therapy safety and to reduce uncertainty that could impact development timelines.